title: Efficacy and safety of growth hormone replacement therapy in Japanese adults with growth hormone deficiency: a post-marketing observational study.
abstract: This large-scale observational study examined the long-term effectiveness and safety of growth hormone (GH) replacement therapy for adult GH deficiency (GHD) in Japanese clinical practice using the Hypopituitary Control and Complications Study database. The study included 402 GHD patients for safety analyses and a subset of 209 patients (149 adult-onset and 60 childhood-onset GHD patients) who had not previously received GH replacement therapy for the efficacy analyses. Data on clinical, metabolic, quality of life (QoL) characteristics, and all adverse events (AEs) were collected at baseline (start of GH treatment), 6 months, 1 year and 2 years. Over the observation period, there were improvements from baseline in insulin-like growth factor-I standard deviation scores (P<0.001), although the changes in metabolic parameters were minimal. QoL (Short Form-36) Z-scores significantly increased from baseline in both onset-type groups for several subscale domains (P<0.05). A total of 145 (36.1%) patients experienced ≥1 AE. Common AEs were hyperlipidaemia (2.7%) and hyperinsulinaemia (2.2%). Some patients experienced recurrent hypothalamic/pituitary tumour (events per 1000 patient-years: 2.78), new benign (0.93), malignant tumour (10.28) or other new tumour (0.93), new diabetes mellitus (7.45), and new stroke (3.71). Seven patients died during the observation period. Our safety findings are inconclusive about the associations between GH replacement and AEs, although the incidence of diabetes mellitus and cardiovascular events are similar to those reported in the Japanese general population. In conclusion, the key beneficial effects of GH replacement therapy for GHD are observed in routine clinical practice in Japan.
authors.affiliation: Clinical Research Institute, National Hospital Organization Kyoto Medical Center, Kyoto 612-8555, Japan.
authors.forename: Akira
authors.initials: A
authors.lastname: Shimatsu
authors.forename: Shigeru
authors.initials: S
authors.lastname: Tai
authors.forename: Makoto
authors.initials: M
authors.lastname: Imori
authors.forename: Katsuichiro
authors.initials: K
authors.lastname: Ihara
authors.forename: Masanori
authors.initials: M
authors.lastname: Taketsuna
authors.forename: Jumpei
authors.initials: J
authors.lastname: Funai
authors.forename: Toshiaki
authors.initials: T
authors.lastname: Tanaka
authors.forename: Akira
authors.initials: A
authors.lastname: Teramoto
authors.forename: Minoru
authors.initials: M
authors.lastname: Irie
authors.forename: Kazuo
authors.initials: K
authors.lastname: Chihara
mesh_terms: D000293:Adolescent; D000328:Adult; D002318:Cardiovascular Diseases / Q000139:chemically induced; D003920:Diabetes Mellitus / Q000139:chemically induced; D005260:Female; D020249:Hormone Replacement Therapy / Q000009:adverse effects; D019382:Human Growth Hormone / Q000009:adverse effects / Q000172:deficiency / Q000627:therapeutic use*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D007564:Japan; D008297:Male; D008875:Middle Aged; D009369:Neoplasms / Q000139:chemically induced; D011788:Quality of Life
chemical_list: D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I